GB201504124D0 - Oligonucleotides - Google Patents

Oligonucleotides

Info

Publication number
GB201504124D0
GB201504124D0 GBGB1504124.7A GB201504124A GB201504124D0 GB 201504124 D0 GB201504124 D0 GB 201504124D0 GB 201504124 A GB201504124 A GB 201504124A GB 201504124 D0 GB201504124 D0 GB 201504124D0
Authority
GB
United Kingdom
Prior art keywords
oligonucleotides
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
GBGB1504124.7A
Other languages
English (en)
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Wings Therapeutics Inc
Original Assignee
ProQR Therapeutics BV
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ProQR Therapeutics BV filed Critical ProQR Therapeutics BV
Priority to GBGB1504124.7A priority Critical patent/GB201504124D0/en
Publication of GB201504124D0 publication Critical patent/GB201504124D0/en
Priority to CN201680015142.2A priority patent/CN107567498B/zh
Priority to CA2978375A priority patent/CA2978375A1/en
Priority to NZ736083A priority patent/NZ736083B2/en
Priority to HUE16709465A priority patent/HUE050898T2/hu
Priority to BR112017019225-0A priority patent/BR112017019225A2/pt
Priority to PL16709465T priority patent/PL3268474T3/pl
Priority to DK16709465.5T priority patent/DK3268474T3/da
Priority to ES16709465T priority patent/ES2803651T3/es
Priority to AU2016231067A priority patent/AU2016231067B2/en
Priority to SI201630794T priority patent/SI3268474T1/sl
Priority to EA201791986A priority patent/EA035732B1/ru
Priority to JP2017547468A priority patent/JP6929783B2/ja
Priority to MX2017011599A priority patent/MX2017011599A/es
Priority to PCT/EP2016/055360 priority patent/WO2016142538A1/en
Priority to KR1020177025038A priority patent/KR20170120617A/ko
Priority to PT167094655T priority patent/PT3268474T/pt
Priority to EP16709465.5A priority patent/EP3268474B1/en
Priority to US15/554,420 priority patent/US10563198B2/en
Priority to EP20175344.9A priority patent/EP3763816A1/en
Priority to IL254173A priority patent/IL254173B/en
Priority to MX2021009135A priority patent/MX2021009135A/es
Priority to US16/746,403 priority patent/US11352626B2/en
Priority to HRP20200944TT priority patent/HRP20200944T1/hr
Priority to JP2021131294A priority patent/JP7188810B2/ja
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
GBGB1504124.7A 2015-03-11 2015-03-11 Oligonucleotides Ceased GB201504124D0 (en)

Priority Applications (25)

Application Number Priority Date Filing Date Title
GBGB1504124.7A GB201504124D0 (en) 2015-03-11 2015-03-11 Oligonucleotides
EP20175344.9A EP3763816A1 (en) 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy
JP2017547468A JP6929783B2 (ja) 2015-03-11 2016-03-11 表皮水疱症治療のためのｃｏｌ７ａ１エクソン７３とマッチするオリゴヌクレオチド
PCT/EP2016/055360 WO2016142538A1 (en) 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy
NZ736083A NZ736083B2 (en) 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy
HUE16709465A HUE050898T2 (hu) 2015-03-11 2016-03-11 Oligonukleotidok, amelyek megfelelnek a COL7A1 73-as exonnak, epidermolysis bullosa terápiához
BR112017019225-0A BR112017019225A2 (pt) 2015-03-11 2016-03-11 oligonucleotídeos compatíveis com o éxon 73 de col7a1 para terapia de epidermólise bolhosa
PL16709465T PL3268474T3 (pl) 2015-03-11 2016-03-11 Oligonukleotydy pasujące do eksonu 73 w COL7A1 do leczenia pęcherzowego oddzielania się naskórka
DK16709465.5T DK3268474T3 (da) 2015-03-11 2016-03-11 Oligonukleotider svarende til exon 73 i col7a1 til epidermolysis bullosa-terapi
ES16709465T ES2803651T3 (es) 2015-03-11 2016-03-11 Emparejamiento de oligonucleótidos con el exón 73 de col7a1 para la terapia de la epidermólisis ampollosa
AU2016231067A AU2016231067B2 (en) 2015-03-11 2016-03-11 Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy
SI201630794T SI3268474T1 (sl) 2015-03-11 2016-03-11 Oligonukleotidi, ki ustrezajo COL7A1 ekson 73 za zdravljenje bulozne epidermolize
EA201791986A EA035732B1 (ru) 2015-03-11 2016-03-11 Олигонуклеотиды, соответствующие экзону 73 гена col7a1, для терапии буллезного эпидермолиза
CN201680015142.2A CN107567498B (zh) 2015-03-11 2016-03-11 用于大疱性表皮松解症治疗的与col7a1外显子73配对的寡核苷酸
MX2017011599A MX2017011599A (es) 2015-03-11 2016-03-11 Oigonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.
CA2978375A CA2978375A1 (en) 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy
KR1020177025038A KR20170120617A (ko) 2015-03-11 2016-03-11 수포성 표피 박리증 치료를 위한 col7a1 엑손 73에 대응하는 올리고뉴클레오타이드
PT167094655T PT3268474T (pt) 2015-03-11 2016-03-11 Oligonucleótidos compatíveis com o exão 73 de col7a1 para terapia de epidermólise bolhosa
EP16709465.5A EP3268474B1 (en) 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy
US15/554,420 US10563198B2 (en) 2015-03-11 2016-03-11 Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy
IL254173A IL254173B (en) 2015-03-11 2017-08-27 Oligonucleotides corresponding to exon 73 of col7a1 for the treatment of blistering of the skin
MX2021009135A MX2021009135A (es) 2015-03-11 2017-09-08 Oligonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.
US16/746,403 US11352626B2 (en) 2015-03-11 2020-01-17 Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy
HRP20200944TT HRP20200944T1 (hr) 2015-03-11 2020-06-12 Oligonukleotidi koji odgovaraju col7a1 eksonu 73 za terapiju bulozne epidermolize
JP2021131294A JP7188810B2 (ja) 2015-03-11 2021-08-11 表皮水疱症治療のためのｃｏｌ７ａ１エクソン７３とマッチするオリゴヌクレオチド

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1504124.7A GB201504124D0 (en) 2015-03-11 2015-03-11 Oligonucleotides

Publications (1)

Publication Number Publication Date
GB201504124D0 true GB201504124D0 (en) 2015-04-22

Family

ID=52998751

Family Applications (1)

Application Number Title Priority Date Filing Date
GBGB1504124.7A Ceased GB201504124D0 (en) 2015-03-11 2015-03-11 Oligonucleotides

Country Status (20)

Country Link
US (2) US10563198B2 (ru)
EP (2) EP3268474B1 (ru)
JP (2) JP6929783B2 (ru)
KR (1) KR20170120617A (ru)
CN (1) CN107567498B (ru)
AU (1) AU2016231067B2 (ru)
BR (1) BR112017019225A2 (ru)
CA (1) CA2978375A1 (ru)
DK (1) DK3268474T3 (ru)
EA (1) EA035732B1 (ru)
ES (1) ES2803651T3 (ru)
GB (1) GB201504124D0 (ru)
HR (1) HRP20200944T1 (ru)
HU (1) HUE050898T2 (ru)
IL (1) IL254173B (ru)
MX (2) MX2017011599A (ru)
PL (1) PL3268474T3 (ru)
PT (1) PT3268474T (ru)
SI (1) SI3268474T1 (ru)
WO (1) WO2016142538A1 (ru)

Families Citing this family (7)

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Publication number Priority date Publication date Assignee Title
EP3302497A4 (en) * 2015-06-01 2019-01-16 Sarepta Therapeutics, Inc. EXCLUSION OF EXON INDUCED PAT TECHNOLOGY ANTISENSE IN COLLAGEN TYPE VII
WO2019146621A1 (ja) * 2018-01-25 2019-08-01 国立大学法人大阪大学 ペリオスチンの発現量増加またはスプライシングバリアントの変化を伴う疾患の治療用医薬組成物
GB201902735D0 (en) 2019-02-28 2019-04-17 Proqr Therapeutics Ii Bv Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa
EP4069842A1 (en) 2019-12-02 2022-10-12 Shape Therapeutics Inc. Therapeutic editing
JP2023554180A (ja) * 2020-12-10 2023-12-26 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ジストロフィー性表皮水疱症を治療するための物質および方法。
WO2024080311A1 (ja) * 2022-10-12 2024-04-18 昌和 栗田 遺伝子導入法、遺伝子治療法、及び組織再生法
CN116555349A (zh) * 2023-01-09 2023-08-08 中吉智药(南京)生物技术有限公司 一种腺相关病毒载体及其构建方法与应用

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HUE051875T2 (hu) 2015-06-15 2021-04-28 Tirmed Pharma Ab Egyláncú oligonukleotidok bõrrendellenességek orvosi kezelésében történõ alkalmazásra
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Also Published As

Publication number Publication date
CA2978375A1 (en) 2016-09-15
AU2016231067B2 (en) 2022-04-21
EP3763816A1 (en) 2021-01-13
JP2018509903A (ja) 2018-04-12
AU2016231067A1 (en) 2017-10-26
CN107567498B (zh) 2021-09-21
EP3268474B1 (en) 2020-05-27
US20200399638A1 (en) 2020-12-24
HUE050898T2 (hu) 2021-01-28
BR112017019225A2 (pt) 2018-05-08
EA201791986A1 (ru) 2018-01-31
NZ736083A (en) 2021-08-27
MX2021009135A (es) 2021-09-10
ES2803651T3 (es) 2021-01-28
EP3268474A1 (en) 2018-01-17
US20180216106A1 (en) 2018-08-02
EA035732B1 (ru) 2020-07-31
PT3268474T (pt) 2020-06-17
JP2022000020A (ja) 2022-01-04
KR20170120617A (ko) 2017-10-31
US11352626B2 (en) 2022-06-07
JP7188810B2 (ja) 2022-12-13
US10563198B2 (en) 2020-02-18
PL3268474T3 (pl) 2020-08-24
HRP20200944T1 (hr) 2020-09-18
DK3268474T3 (da) 2020-06-15
CN107567498A (zh) 2018-01-09
SI3268474T1 (sl) 2020-07-31
IL254173B (en) 2022-03-01
WO2016142538A1 (en) 2016-09-15
MX2017011599A (es) 2018-06-15
IL254173A0 (en) 2017-10-31
JP6929783B2 (ja) 2021-09-01

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